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更新于：2025-04-17
Batoclimab
巴托利单抗
更新于：2025-04-17
概要
基本信息
药物类型 单克隆抗体 |
别名 Batoclimab (USAN/INN)、Immunoglobulin g1(238-alanine, 239-alanine), anti-(human fcrn receptor) (human monoclonal hl161bkn .gamma.1-chain), disulfide with human monoclonal hl161bkn .lambda.-chain, dimer、HBM-9161 + [5] |
靶点 |
作用方式 拮抗剂、调节剂 |
作用机制 FcRn拮抗剂(IgG受体FcRn大亚基p51拮抗剂)、免疫调节剂 |
非在研适应症 |
非在研机构- |
最高研发阶段申请上市 |
首次获批日期- |
最高研发阶段(中国)申请上市 |
特殊审评突破性疗法 (中国)、孤儿药 (日本) |
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结构/序列
Sequence Code 524289076H
来源: *****
Sequence Code 524289077L
来源: *****
关联
35
项与 巴托利单抗 相关的临床试验NCT05907668
IMVT-1401-2501: A Proof-of-Concept, Open-label Study to Assess the Safety and Efficacy of Batoclimab in Participants With Graves' Disease (GD)
The purpose of this study is to assess the efficacy and safety of 24 weeks of treatment with batoclimab in adult participants with biochemically documented hyperthyroidism due to GD who have failed to achieve euthyroidism on antithyroid drugs (ATDs).
开始日期2023-05-15 |
申办/合作机构 |
EUCTR2022-002894-27-DE
A Proof-of-Concept, Open-label Study to Assess the Safety and Efficacy of Batoclimab in Participants with Graves’ Disease (GD)
开始日期2023-04-03 |
申办/合作机构 |
NCT05524571
A Phase 3, Multi-center, Randomized, Quadruple-masked, Placebo-controlled Study of Batoclimab for the Treatment of Participants With Active Thyroid Eye Disease (TED)
To evaluate the efficacy of batoclimab 680 milligrams (mg) subcutaneous (SC) once a week (QW) for 12 weeks followed by 340 mg SC QW for 12 weeks versus placebo on proptosis responder rate at Week 24.
开始日期2023-01-05 |
申办/合作机构 |
100 项与 巴托利单抗 相关的临床结果
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100 项与 巴托利单抗 相关的转化医学
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100 项与 巴托利单抗 相关的专利（医药）
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20
项与 巴托利单抗 相关的文献（医药）2024-06-01·CNS DRUGS
FcRn Inhibitor Therapies in Neurologic Diseases
Review
作者: Karmastaji, Salama ; Matic, Alexandria ; Alfaidi, Nouf ; Bril, Vera
In the last decade, the landscape of treating autoimmune diseases has evolved with the emergence and approval of novel targeted therapies. Several new biological agents offer selective and target-specific immunotherapy and therefore fewer side effects, such as neonatal Fc receptor (FcRn)-targeting therapy. Neonatal Fc receptor-targeted therapies are engineered to selectively target FcRn through various methods, such as Fc fragments or monoclonal anti-FcRn antibodies. These approaches enhance the breakdown of autoantibodies by blocking the immunoglobulin G recycling pathway. This mechanism reduces overall plasma immunoglobulin levels, including the levels of pathogenic autoantibodies, without affecting the other immunoglobulin class immunoglobulin A, immunoglobulin E, immunoglobulin M, and immunoglobulin D levels. Drugs that inhibit FcRn include efgartigimod, rozanolixizumab, batoclimab, and nipocalimab. These medications can be administered either intravenously or subcutaneously. Numerous clinical trials are currently underway to investigate their effectiveness, safety, and tolerability in various neurological conditions, including myasthenia gravis and other neurological disorders such as chronic inflammatory demyelinating polyneuropathy, myositis, neuromyelitis optica, and myelin oligodendrocyte glycoprotein antibody disease. Positive results from clinical trials of efgartigimod and rozanolixizumab led to their approval for the treatment of generalized myasthenia gravis. Additional clinical trials are still ongoing. Neonatal Fc receptor inhibitor agents seem to be well tolerated. Reported adverse events include headache (most commonly observed with efgartigimod and rozanolixizumab), upper respiratory tract infection, urinary tract infection, diarrhea, pyrexia, and nausea. Additionally, some of these agents may cause transient hypoalbuminemia and hypercholesterolemia notably reported with batoclimab and nipocalimab. In this review, we discuss the available clinical data for FcRN inhibitor agents in treating different neurological autoimmune diseases.
2024-05-01·Journal of neurology
The efficacy and safety of FcRn inhibitors in patients with myasthenia gravis: a systematic review and meta-analysis
Review
作者: Li, Jiaxuan ; Wu, Xin ; Chu, Tianchen ; Chen, Zhouqing ; Wang, Shixin ; Tan, Xin ; Qu, Ruisi ; Wang, Zhong
BACKGROUND:
Myasthenia gravis (MG) is an autoimmune disease that causes local or generalized muscle weakness. Complement inhibitors and targeting of the neonatal Fc receptor (FcRn) to block IgG cycling are two novel and successful mechanisms.
METHODS:
PubMed, EMBASE, the Cochrane Library, and ClinicalTrials.gov were systematically searched to identify relevant studies published before May 18, 2023. Review Manager 5.3 software was used to assess the data.
RESULTS:
We pooled 532 participants from six randomized controlled trials (RCTs). Compared to the placebo, the FcRn inhibitors were more efficacy in Myasthenia Gravis Activities of Daily Living (MG-ADL) (MD = - 1.69 [- 2.35, - 1.03], P < 0.00001), MG-ADL responder (RR = 2.01 [1.62, 2.48], P < 0.00001), Quantitative Myasthenia Gravis (QMG) (MD = - 2.45 [- 4.35, - 0.55], P = 0.01), Myasthenia Gravis Composite (MGC) (MD = - 2.97 [- 4.27, - 1.67], P < 0.00001), 15-item revised version of the Myasthenia Gravis Quality of Life (MGQoL15r) (MD = - 2.52 [- 3.54, - 1.50], P < 0.00001), without increasing the risk of safety. The subgroup analysis showed that efgartigimod was more effective than placebo in MG-ADL responders. Rozanolixizumab was more effective than the placebo except in QMG, and batoclimab was more effective than the placebo except in MG-ADL responder. Nipocalizumab did not show satisfactory efficacy in all outcomes. With the exception of rozanolixizumab, all drugs showed non-inferior safety profiles to placebo.
CONCLUSION:
FcRn inhibitors have good efficacy and safety in patients with MG. Among them, efgartigimod and nipocalimab were effective without causing an increased safety risk. Rozanolixizumab, despite its superior efficacy, caused an increased incidence of adverse events. Current evidence does not suggest that nipocalimab is effective in patients with MG.
2024-04-01·JAMA neurology
Batoclimab vs Placebo for Generalized Myasthenia Gravis
Article
作者: Ke, Qing ; Tan, Ying ; Zhao, Hongdong ; Zheng, Hui ; Zou, Zhangyu ; Dai, Tingjun ; Fu, Jia ; Dong, Jun ; Jin, Luya ; Duan, Ruisheng ; Li, Haifeng ; Chen, Sheng ; Li, Zunbo ; Luo, Jing ; Huang, Wenjiao ; Zhao, Chongbo ; Li, Xiuyun ; Zhao, Mingming ; Tu, Jianglong ; Zeng, Wenshuang ; Hu, Xingyue ; Ding, Xiaojun ; Xi, Jianying ; Ruan, Zhe ; Chang, Xueli ; Shi, Jiayu ; Yang, Bing ; Zhou, Hongyu ; Hou, Shifang ; Chen, Yu ; Yang, Ruihan ; Zeng, Quantao ; Zhang, Hua ; Xiao, Fei ; Wang, Jianwen ; Deng, Hui ; Lee, Michael ; Zheng, Rui ; Zhang, Yali ; Li, Huanhuan ; Tan, Song ; Jiao, Lidong ; Shi, Jianquan ; Zhang, Min ; Xiao, Haibing ; Yan, Chuanzhu ; Xiao, Xingyi ; Zhang, Wei ; Zhao, Yuying ; Jiang, Haishan ; Xiong, Ting ; Tao, Xiaolu ; Meng, Qiang ; Chen, Jialin ; Zhou, Hao ; Zhang, Lei ; Guan, Yuzhou ; Lu, Hui ; Song, Min ; Li, Ping ; Li, Wei ; Chang, Ting ; Du, Qin ; Ji, Xiaopei ; Ma, Jiaojiao ; Bai, Xue ; Chen, Kai ; Guo, Junhong ; Zeng, Li ; Li, Wenyu ; Wang, Congcong ; Xue, Qun ; Zhao, Shuai ; Yue, Yaoxian ; Bu, Bitao ; Li, Xiaoli ; Sun, Hongbin ; Zhang, Meng ; Xie, Yan ; Yan, Chong ; Yang, Huan ; Zhang, Bin ; Yin, Jian ; Zhao, Cuiping ; Wang, Xu
Importance:
Myasthenia gravis (MG) is caused by autoantibodies that disrupt the neuromuscular junction. The neonatal fragment crystallizable receptor (FcRn) antagonists, efgartigimod and rozanolixizumab, reduce immunoglobulin G (IgG) level in the circulation and alleviate symptoms in patients with generalized MG.
Objective:
To examine the efficacy and safety profile of batoclimab, a monoclonal IgG1 antibody, in patients with generalized MG.
Design, Setting, and Participants:
This was a multicenter randomized clinical trial conducted from September 15, 2021, to June 29, 2022, at 27 centers in China. Adult patients 18 years or older with generalized MG were screened, and those who were antibody positive were enrolled.
Intervention:
Eligible patients received batoclimab or matching placebo in addition to standard of care. Each treatment cycle consisted of 6 weekly subcutaneous injections of batoclimab, 680 mg, or matching placebo followed by 4 weeks of observation. A second treatment cycle was conducted in patients who required continuing treatment.
Main Outcome and Measure:
The primary outcome was sustained improvement, as defined by a 3-point or greater reduction in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score from baseline for 4 or more consecutive weeks in the first cycle in individuals who were positive for acetylcholine receptor or muscle-specific kinase antibodies.
Results:
A total of 178 adult patients with generalized MG were screened, 132 were randomly assigned, 131 tested positive for antibodies, and 1 tested negative for antibodies. A total of 132 patients (mean [SE] age, 43.8 [13.6] years; 88 women [67.2%]) were enrolled. The rate of sustained MG-ADL improvement in the first cycle in antibody-positive patients was 31.3% (20 of 64) in the placebo group vs 58.2% (39 of 67) in the batoclimab group (odds ratio, 3.45; 95% CI, 1.62-7.35; P = .001). The MG-ADL score diverged between the 2 groups as early as week 2. The mean (SE) maximum difference in MG-ADL score reduction occurred 1 week after the last dose (day 43, 1.7 [0.3] in the placebo group vs 3.6 [0.3] in the batoclimab group; group difference, −1.9; 95% CI, −2.8 to −1.0; nominal P &lt; .001). The rates of treatment-related and severe treatment-emergent adverse events in patients were 36.9% (24 of 65) and 7.7% (5 of 65) in the placebo group vs 70.1% (47 of 67) and 3.0% (2 of 67) in the batoclimab group, respectively.
Conclusions and Relevance:
Batoclimab increased the rate of sustained MG-ADL improvement and was well tolerated in adult patients with generalized MG. Clinical effects and the extent of IgG reduction were similar to those previously reported for efgartigimod and rozanolixizumab. Future studies of large sample size are needed to further understand the safety profile of batoclimab.
Trial Registration:
ClinicalTrials.gov Identifier: NCT05039190
335
项与 巴托利单抗 相关的新闻（医药）2025-04-11
作者｜小麦近日，全球知名医药数据公司Evaluate Pharma对2025年医药行业发展趋势发表见解，其中包括对一些Biopharma的核心资产进行预测，特别是关注到Biopharma都在押宝什么？（图1）图1. Biopharma的十大潜在重磅核心资产从这些管线中，我们可以看出在过去的几年里，生物制药公司已经将大量注意力转移到免疫学上，近期又转向了代谢领域，尤其是司美格鲁肽的爆火引爆了GLP-1类减肥药的研发。除了司美格鲁肽，诺和诺德、礼来和安进都把宝押在GLP-1类药物上，包括诺和诺德的复方减肥药Cagrisema、礼来的三重激动剂Retatrutide、小分子GLP-1R激动剂Orforglipron以及安进的每月一次给药的MariTide。Immunovant/Roivant把希望寄托在靶向FcRn（新生儿 Fc 受体）的药物IMVT-1402和Batoclimab，它们是一类新型的免疫调节药物，主要用于治疗 IgG 介导的自身免疫性疾病，IMVT-1402有望成为Best-in-class的靶向FcRn药物。在Evaluate Pharma所关注到核心管线中，只有一款是用于治疗肿瘤的，即康方生物靶向PD-1xVEGF 双抗药物Ivonescimab。2022年底，康方生物与 Summit Therapeutics就Ivonescimab 的全球开发和商业化达成高达 50 亿美元的合作和许可协议，由此可见Ivonescimab的价值无限。除此之外，还有两款RNA类药物，包括安进的靶向脂蛋白(a)（Lp(a)）的小干扰 RNA（siRNA）药物Olpasiran、Moderna/Merck的一种基于 mRNA 的个性化新抗原疗法mRNA-4157以及一款自免药物即Moonlake的Sonelokimab。 01 诺和诺德：Cagrisema诺和诺德在GLP-1类减肥药领域取得巨大成功，其重磅产品司美格鲁肽（Semaglutide）在2024年的总销售额达到了2018.49亿丹麦克朗，约合293亿美元，占据诺和诺德公司2024年总营收的约69.5%。除此之外，诺和诺德积极布局寻找下一个强力增长点，他们把希望放在了CagriSema身上。CagriSema是由司美格鲁肽和胰淀素类似物Cagrilintide组成的一种新型复方减肥药，司美格鲁肽能够刺激胰岛素的生成，抑制胰高血糖素分泌，延缓胃排空，并通过中枢性的食欲抑制来减少进食量，从而降低血糖和减轻体重，胰淀素是一种与饥饿和饱腹感相关的激素，也能参与胰岛素的调节，两种药物通过减少饥饿感、增加饱腹感，来减少食物和热量的摄入，从而降低体重。此前，CagriSema的一项临床2期结果显示：CagriSema注射液（司美格鲁肽2.4mg和Cagrilintide 2.4mg组成）组患者的HbA1c水平降低了2.18%，体重减轻了15.6%，减重和降糖效果均优于司美格鲁肽2.4mg组或cagrilintide 2.4mg组（图2）。图2. CagriSema的临床2期结果自去年年底至今，诺和诺德相继公布了CagriSema的两项3期研究结果，包括REDEFINE 1和REDEFINE 2。2024年12月，诺和诺德宣布了全球REDEFINE计划的3期试验 REDEFINE 1 的主要结果：安慰剂相比，CagriSema在第68周时的体重减轻具有统计学意义且更好，从而达到了其主要终点。具体来说，在评估所有受试者都坚持治疗的情况下的治疗效果时，接受 CagriSema 治疗的患者在68周后实现了22.7%的体重减轻，而2.4 mg cagrilintide治疗组减少了11.8%，司美格鲁肽2.4 mg组减少了16.1%，单独使用安慰剂组减少了2.3%。此外，40.4%接受CagriSema治疗的患者在68周后体重减轻了25% 或更多，而 cagrilintide 2.4 mg组为6.0%，司美格鲁肽2.4 mg组为16.2%，安慰剂组为0.9%（图3）[1,2]。图3. REDEFINE 1试验结果2025年3月10日，诺和诺德宣布了CagriSema的另一项全球 REDEFINE 计划的3期试验REDEFINE 2的主要结果：在评估治疗效果时，如果所有人都坚持治疗，则接受 CagriSema 治疗的人在68周后实现了15.7%的体重减轻，而安慰剂组为3.1%。68周后体重减轻5%或以上是共同主要终点，89.7%的 CagriSeama患者实现了体重减轻，而安慰剂组为30.3%（图4）。图4. REDEFINE 2试验结果然而虽然CagriSema的两项3期试验都取得积极结果，但是都不达预期，这也导致近期诺和诺德股价大跌。 02 礼来：Retatrutide和OrforglipronRetatrutide 是礼来开发的一种靶向GLP-1、葡萄糖依赖性促胰岛素多肽（GIP）和胰高血糖素（GCG）的由39个氨基酸组成的三重受体激动剂，这些受体中的每一种都在调节新陈代谢中起着重要作用，特别关注胰岛素分泌、能量消耗和血糖水平（图5）[3]。图5. Retatrutide作用机制Retatrutide 的首次人体试验于2019年在新加坡进行。这项1期概念验证研究涉及47名参与者，每周接受0.1 mg至6 mg的retatruide。研究结果表明，Retatrutide对食欲调节、食物摄入和代谢标志物有积极的影响，同时该研究还显示了了retatrutide的良好安全性[4]。Retatrutide 的一项2期研究结果显示：在48周时，retatrutide在1 mg组中使受试者体重下降8.7%，在联合 4 mg 组下降17.1%，8 mg联合组下降22.8%，12 mg组下降24.2%，而安慰剂组下降2.1%（图6）[5]。图6. Retatrutide 的2期研究结果目前Retatrutide正在开展多项TRIUMPH 3期临床试验，旨在评估Retatrutide在肥胖或超重患者中的安全性和有效性，包括TRIUMPH 1-4等（图7）[6]。图7. Retatrutide的临床试验除此之外，Retatrutide还在开展TRANSCEND系列3期试验，预计明年至后年完成（图8）。图8. Retatrutide的临床试验Orforglipron是由Chugai开发的一种可口服的小分子GLP-1R激动剂, 每日服用一次，主要用于治疗肥胖症和2型糖尿病。2018年9月，礼来与中外制药达成许可协议，以5000万美元首付款获得该产品的全球开发权和商业化权。临床前研究表明，Orforglipron降糖效果与艾塞那肽相当。在食蟹猴中进行的药代动力学实验结果显示，Orforglipron的半衰期为3.4~4.6h，生物利用度达到21~28%（图9）。图9. Orforglipron的结构与活性Orforglipron目前正在临床上开展多项试验，包括针对肥胖症的3期ATTAIN-1和ATTAIN-2试验、针对2型糖尿病患者的3期临床试验以及针对阻塞性睡眠呼吸暂停的3期临床试验等（图10和图11）。图10. Orforglipron的临床试验图11. Orforglipron的临床试验此前针对肥胖症的2期临床研究显示，接受高剂量Orforglipron治疗的肥胖或超重成人在36周后平均体重减轻了14.7%。针对2型糖尿病患者2期临床试验结果显示，经过26周的治疗后，45mg 剂量组患者的糖化血红蛋白（HbA1c）平均降幅达到2.1%，体重平均下降 10.1kg。礼来计划在2025年在美国、欧盟、日本递交 Orforglipron 的上市申请。如果一切顺利，Orforglipron最快有望在2026年获批上市。 03 安进：MariTideMariTide是由安进（Amgen）开发的一种新型双特异性分子药物，属于抗体-多肽偶联物（Peptibody）。与其它GLP-1类激动剂不同，它是葡萄糖依赖性促胰岛素多肽受体（GIPR）拮抗剂和胰高血糖素样肽-1（GLP-1）受体激动剂（图12）。与传统的GLP-1受体激动剂（如司美格鲁肽）和GIPR激动剂（如替尔泊肽）不同，MariTide是首个将GLP-1激动剂与GIPR拮抗剂结合的药物。GLP-1 受体激动剂能够刺激胰岛素分泌，抑制食欲；而 GIPR 拮抗剂则通过抑制 GIP 的作用，减少胰岛素分泌，从而降低食欲和体重。图12. MariTide作用机制2024年11月26日，安进公司宣布了一项双盲、剂量探索的2期研究 MariTide的52周阳性数据：在没有2型糖尿病的肥胖或超重患者中，MariTide 在第52周时平均体重减轻了高达~20%，没有体重减轻平台期，这表明52周后体重可能会进一步减轻（图13）[7]。该研究还显示，肥胖或超重患者以及 2 型糖尿病患者通常在 GLP-1 疗法中减轻的体重较少。平均体重减轻高达~17%，也没有体重减轻平台期，并在第52.1周将平均血红蛋白A1C（HbA1c）降低高达2.2%，总之，在两个研究人群中，均未观察到体重减轻平台期，再次表明52周后体重进一步减轻的可能性。图13. MariTide基本信息 04 Immunovant/Roivant：IMVT-1402和BatoclimabIMVT-1402是一种由Immunovant公司开发的潜在“best-in-class”新生儿 Fc受体（FcRn）靶向抗体，主要用于治疗IgG介导的自身免疫性疾病（图14）[8-11]。图14. IMVT-1402基本信息FcRn是一种免疫球蛋白G（IgG）和白蛋白的特异性受体，由MHC I类相关分子和β2微球蛋白组成。它通过酸碱度依赖的方式与IgG和白蛋白结合，使它们免于被溶酶体降解，从而延长其在血浆中的半衰期。此外，FcRn还具有实现IgG和白蛋白的跨膜转运及促进抗原提呈的作用（图15）。图15. FcRn作用FcRn与多种IgG介导的自身免疫性疾病密切相关，包括重症肌无力（MG）、慢性炎症性脱髓鞘性多发性神经病（CIDP）、甲状腺眼病（TED）、免疫性血小板减少症（ITP）以及格雷夫斯病（Graves’ Disease）等（图16）。 图16. FcRn与多种IgG介导的自身免疫性疾病密切相关在1期临床试验中，IMVT-1402表现出剂量依赖性地显著降低IgG水平。在300mg多剂量组中，经过4周的皮下注射，平均IgG降低63%；在600mg 多剂量组中，经过4次皮下注射，平均IgG降低74%（图17）。图17. IMVT-1402的1期试验结果IMVT-1402在格雷夫斯病中显示出“Best and First-in-class”特征。与 Batoclimab对比，IMVT-1402的IgG降低效果与Batoclimab类似，Batoclimab在高剂量（680 mg）下可使 IgG 降低 76%（图18）。图18. IMVT-1402在格雷夫斯病中显示出“Best and First-in-class”特征目前IMVT-1402正处于临床2期，正在开展针对类风湿关节炎和格雷夫斯病的临床试验，同时准备开展IMVT-1402在重症肌无力（MG）和慢性炎症性脱髓鞘性多发性神经病（CIDP）等4至5种适应症的临床项目，并计划在次年拓展至10种适应症（图19）。图19. IMVT-1402的临床试验计划Batoclimab（RVT-1401/HL161）是由Immunovant开发的一款早于IMVT-1402的靶向FcRn的一种全人源IgG1单克隆抗体，通过阻断FcRn与IgG的结合来降低致病性IgG水平。近期，Immunovant公布了Batoclimab治疗MG的3期研究的顶线结果，以及CIDP的2b期研究的第1期初步结果。Batoclimab治疗MG的3期研究结果显示：Batoclimab组在12周时达到了AChR人群中MG-ADL相对于基线变化的主要终点，其中高剂量组提高了5.6分（平均IgG减少74%），低剂量组提高了4.7分（平均 IgG 减少 64%）（图20）。图20. Batoclimab治疗MG的3期研究结果Batoclimab治疗CIDP的2b期研究的第1期初步结果：Batoclimab组调整后的 INCAT 残疾评分平均改善1.8，IgG 降低超过70%的患者反应率为 84%（图21）。图21. Batoclimab治疗CIDP的2b期研究的第1期初步结果在这两项 batoclimab 研究中，IgG 减少的程度更深与一系列评估和时间点的更好临床结果相关。 05 Moonlake Immunotherapeutics：SonelokimabSonelokimab（SLK，M1095）是由MoonLake Immunotherapeutics研发的一种~40 kDa人源化纳米抗体，由三个VHH结构域组成，这些结构域通过灵活的甘氨酸-丝氨酸间隔区共价连接。Sonelokimab 的两个结构域，以高亲和力选择性结合IL-17A和IL-17F，从而抑制 IL-17A/A、IL-17A/F和IL-17F/F二聚体，从而抑制炎症。第三个中心结构域与人白蛋白结合，促进sonelokimab在炎性水肿部位的进一步富集（图22）[12]。图22. Sonelokimab作用机制Sonelokimab正在评估化脓性汗腺炎（HS）和银屑病关节炎（PsA）这两个主要适应症，同时，MoonLake也在寻求皮肤病学和风湿病学的其他适应症（图23）。今年1月8日，MoonLake宣布，已在青少年化脓性汗腺炎 （HS）、掌跖脓疱病 （PPP） 和中轴型脊柱关节炎 （axSpA） 患者三个新适应症的三项新试验中筛选了患者，以评估 sonelokimab，一种旨在治疗炎症性疾病的研究性纳米抗体®。这将使 MoonLake 主导的临床试验数量增加到 10 项，并与自 2021 年公司成立以来招募 3,000 多名患者参加 sonelokimab 临床试验的计划保持一致。图23. Sonelokimab临床试验2023 年 6 月，sonelokimab 的2期MIRA 试验 （NCT05322473） 在 12 周时的顶线结果显示，该试验达到了其主要终点，即化脓性汗腺炎临床反应 （HiSCR）75，与其他临床试验中使用的 HiSCR50 指标相比，该指标的临床反应指标更高，树立了一个里程碑（图24）。图24. Sonelokimab的MIRA试验结果2023年10月，MIRA 试验在24周时的完整数据集显示，sonelokimab 维持治疗进一步改善了HiSCR75反应率和其他高阈值临床和患者相关结局（图25）。sonelokimab 在 MIRA 试验中的安全性特征与之前的试验一致，未检测到新的安全性信号。图25. Sonelokimab的MIRA 试验24周时的完整数据2024年2月，MoonLake宣布FDA和EMA对sonelokimab治疗化脓性汗腺炎（HS）的3期试验的监管路径给予积极反馈，基于此，MoonLake开展了3期VELA-1 和 VELA-2临床试验。对于 PsA，继2024年3月宣布全球2期ARGO试验（M1095-PSA-201） 的完整数据集后，sonelokimab正在3期试验 IZAR-1和IZAR-2中评估在24周内对活动性PsA患者的疗效和安全性（图26）。图26. Sonelokimab的 3期试验 IZAR方案Sonelokimab的ARGO试验结果显示在所有关键结局中均观察到显著改善，包括约60%的sonelokimab治疗患者在第24周达到美国风湿病学会（ACR）50 缓解和最小疾病活动度（MDA）（图27）。注：ACR 50 是美国风湿病学会（ACR）制定的用于评估类风湿关节炎（RA）患者治疗反应的标准之一。它表示患者在治疗后，疾病活动度相比基线改善了 50% 或以上。图27. Sonelokimab的ARGO试验结果这是继 2023 年 11 月的积极顶线结果之后，该试验达到了主要终点，与安慰剂组相比，接受 sonelokimab 60mg 或 120mg（诱导）治疗的患者在第 12 周达到 ACR50 反应的比例具有统计学意义。试验中的所有关键次要终点均达到 60 mg和 120 mg诱导剂量[13]。 小结 今年Evaluate Pharma对Biopharma的未来潜力管线进行了预测，其中大部分药物都是治疗代谢疾病的，主要是GLP-1类药物，有三款免疫药物，即IMVT-1402、Batoclimab和Sonelokimab，另有两款RNA类药物，包括Olpasiran和 mRNA-4157以及一款肿瘤药物Ivonescimab。一款药物的成功不仅使患者受益，也能使企业得到丰厚的回报。 主要参考文献 1. Novo Nordisk A/S: CagriSema demonstrates superior weight loss in adults with obesity or overweight in the REDEFINE 1 trial2. Novo Nordisk– a focused healthcare company Investor presentation Full year 20243. The power of three: Retatrutide's role in modern obesity and diabetes therapy, European Journal of Pharmacology Volume 985, 15 December 2024, 1770954. Retatrutide | Reviews, Clinical Data, Benefits & Side Effects5. Triple–Hormone-Receptor Agonist Retatrutide for Obesity — A Phase 2 Trial, N Engl J Med 2023;389:514-266. ELI LILLY AND COMPANY Q4 2024 EARNINGS CALL 02.06.257. AMGEN ANNOUNCES ROBUST WEIGHT LOSS WITH MARITIDE IN PEOPLE LIVING WITH OBESITY OR OVERWEIGHT AT 52 WEEKS IN A PHASE 2 STUDY8. Immunovant Development Update9. Targeted science, Tailored solutions for people with autoimmune disease2025 J.P. Morgan Healthcare Conference January 14, 2025，immunovant10. Targeted science, Tailored solutions for people with autoimmune disease MG & CIDP Results March 202511. Immunovant Announces Positive Results for Batoclimab Myasthenia Gravis (MG) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Studies12. MoonLake Immunotherapeutics October 2024 Corporate Presentation13. MoonLake initiates three new clinical trials and further expands the portfolio of indications for the Nanobody® sonelokimab共建Biomedical创新生态圈！如何加入BiG会员？
siRNA临床2期财报
2025-04-09
The funding was co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock Ventures. Credit: Dalin Ou via Getty Images.
US biotech Merida Biosciences has launched with $121m in Series A financing to develop therapeutics targeting disease-causing antibodies in autoimmune diseases and allergies.
The financing round was co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock Ventures, with participation from GV and Perceptive Xontogeny Venture (PXV) Funds.
The biotech is advancing a proprietary protein engineering platform to develop fragment crystallisable (Fc)-based biologics that selectively bind and eliminate pathogenic antibodies while sparing healthy components of the immune system. Merida’s therapeutics also aim to deplete the B cells responsible for producing the harmful antibodies.
Merida’s lead programme will focus on Graves’ disease, an
autoimmune condition
that causes the thyroid to overproduce hormones, resulting in hyperthyroidism. The company is positioning its candidate as a more targeted alternative to antithyroid drugs (ATDs), which while being standard of care (SOC), often fail to provide adequate disease control.
The company claims its approach could offer a more convenient dosing schedule and an easier route of administration compared to current and emerging treatments.
Merida is not the only biotech setting its sights on the Graves’ disease market. Immunovant is developing batoclimab, a monoclonal antibody
currently in a Phase IIa trial
(NCT05907668) for Graves’ disease patients who remain uncontrolled on ATDs. Interim data released in September 2024 showed a 76% response rate at 12 weeks. If batoclimab makes it to market, GlobalData projects that it will generate $872m in global sales by 2030.
GlobalData is the parent company of
Pharmaceutical Technology.
In addition to Merida and Immunovant, Biohaven is developing BHV-1300, a small molecule therapy that targets and degrades immunoglobulin G1 (IgG1) autoantibodies that cause Graves’ disease. BioHaven previously announced plans to launch a Phase II study of the drug in mid-2025.
Following its lead Graves’ disease programme, Merida plans to pursue candidates for food allergies and primary membranous nephropathy, a rare autoimmune kidney disorder.
Merida will be led by CEO Adam Townsend, former COO of Apellis Pharmaceuticals and ex-Biogen executive. He will work alongside chief scientific officer Dario Gutierrez, a former executive director at MSD and current consultant at Third Rock Ventures.
“For the first time, we have the potential to precisely target the clear pathogenic drivers of an intractable set of diseases with absolute selectivity and a degree of completeness and durability that has not been achievable through any approach to date,” said Townsend in the 8 April announcement.
临床2期
2025-04-08
Immunology continues to be one of the most active areas for drug research as the field aims to bring patients new ways to eliminate the antibodies driving autoimmune disease, but in a manner that does not also compromise the immune system. Merida Biosciences, a startup formed by venture capital firm Third Rock Ventures, emerged from stealth Tuesday, revealing $121 million in financing and a lead program for a common autoimmune condition that has not had a new approved therapy in decades.
The lead disease target of Cambridge, Massachusetts-based Merida is Graves’ disease, which develops as antibodies produced by the immune system attack the thyroid gland, a butterfly-shaped gland located at the front of the neck. Thyroid hormone regulates functions such as metabolism and growth. In Graves’ disease, the attack on the gland results in excess thyroid hormone production that speeds up the heart rate and weakens bones among other problems.
Standard Graves’ treatment includes radioactive iodine therapy to shrink the gland, anti-thyroid medications, or beta blockers that block the effect of thyroid hormone on the body. Each of these treatments introduces complication risks elsewhere in the body. Surgical removal of the thyroid gland is another treatment option, but patients need lifelong hormone replacement therapy afterward.
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Merida is developing drugs designed to selectively bind to Fc receptors, receptors on immune cells that regulate immune responses. The company says its protein engineering platform generates unique Fc therapeutics that selectively bind to their antibody targets. The drugs then lead to the elimination of these antibodies by leveraging built-in mechanisms for clearing antibody complexes, which are molecular structures formed when an antibody binds to its specific target antigen. The company says its drugs are designed to eliminate only pathogenic antibodies, sparing healthy components of the immune system. In Graves’ disease, this approach is intended to spare healthy thyroid signaling.
The Merida drugs are biotherapeutics that the startup describes as “antibody-like” in the way they move through the body. For example, these drugs could provide longer-lasting effects that enable less frequent dosing compared to some other therapies. The Graves program is currently undergoing the preclinical research that could support an investigational new drug application. Other disclosed Merida programs are in preclinical development for primary membranous nephropathy, which is a chronic inflammatory kidney disease, and food allergies mediated by immunoglobulin E antibodies.
“At the heart of Merida is understanding the mechanisms behind antibody-driven diseases like Graves’ at the molecular level, and leveraging these insights to develop precision therapeutics that have thus far evaded the field,” Merida co-founder and Chief Scientific Officer Dario Gutierrez said in a prepared statement. “Our highly differentiated approach has the potential to yield best-in-class treatments by going after the root causes of these devastating diseases.”
Merida’s launch comes as other immunology-focused biotech companies, including startups, make headway with their own selective Fc-receptor targeting drugs. Roivant Sciences subsidiary Immunovant is pursuing various immunological indications with IMVT-1402 and batoclimab. Both drug candidates are monoclonal antibodies. The clinical program for IMVT-1402 includes a Phase 2/3 study underway in Graves’. Batoclimab is currently in Phase 2 testing in Graves’. Last September, Immunovant reported preliminary data showing batoclimab achieved a strong response and a lowering of levels of antibodies associated with the disease. The study is expected to post additional data this summer.
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Zenas Biopharma is developing bifunctional antibodies for immunology and inflammation indications. Lead program obexelimab is designed to bind to CD19 and Fc gamma receptor IIb, both of them targets expressed on B cells. A Phase 3 test is underway in immunoglobulin G4-related disease, supported by a $225 million IPO last September. Meanwhile, startup Nuvig Therapeutics is developing proteins engineered to target Fc receptors. Late last year, the Nuvig raised $161 million to support mid-stage clinical development of its lead program, a potential treatment for the rare autoimmune condition chronic inflammatory demyelinating polyneuropathy.
Merida received seed financing from Third Rock in 2022, when Gutierrez was an entrepreneur in residence at the firm. The Series A financing announced Tuesday was co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock. The other investors in the new round are GV and Perceptive Xontogeny Venture Funds. The startup is led by CEO Adam Townsend, who was most recently chief operating officer at Apellis Pharmaceuticals, a company that develops drugs that address the complement system, a part of the immune system.
Illustration: Sebastian Kaulitzki/Science Photo Library, via Getty Images
临床2期IPO
100 项与 巴托利单抗 相关的药物交易
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 重症肌无力 | 申请上市 | 中国 | 2023-06-28 | |
| 重症肌无力 | 申请上市 | 中国 | 2023-06-28 | |
| 重症肌无力 | 申请上市 | 中国 | 2023-06-28 | |
| 免疫性血小板减少症 | 临床3期 | 中国 | 2022-06-23 | |
| 格雷夫斯眼病 | 临床3期 | 中国 | 2021-09-22 | |
| 血小板减少症 | 临床3期 | - | - | |
| 血小板减少症 | 临床3期 | - | - | |
| 慢性炎症性脱髓鞘性多发性神经病 | 临床2期 | 美国 | 2022-12-15 | |
| 慢性炎症性脱髓鞘性多发性神经病 | 临床2期 | 阿根廷 | 2022-12-15 | |
| 慢性炎症性脱髓鞘性多发性神经病 | 临床2期 | 比利时 | 2022-12-15 |
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临床结果
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适应症
分期
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| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床3期 | 重症肌无力 acetylcholine receptor antibody positive (AChR+) | - | Batoclimab 340mg weekly | 齋醖積鹽簾齋範膚選壓(願鬱淵醖襯積鏇憲鑰餘) = 壓蓋構築衊網夢觸觸壓 窪廠醖廠糧網鬱衊鹽餘 (夢繭淵壓餘壓範淵簾鬱 ) 达到 更多 | 积极 | 2025-03-19 | |
Batoclimab 680mg weekly | 齋醖積鹽簾齋範膚選壓(願鬱淵醖襯積鏇憲鑰餘) = 願鹽簾鏇築積築夢鹹夢 窪廠醖廠糧網鬱衊鹽餘 (夢繭淵壓餘壓範淵簾鬱 ) 达到 更多 | ||||||
临床2期 | 73 | Batoclimab 340 mg/680 mg | 網獵蓋夢選蓋鹹簾襯鑰(醖壓鬱選製範鑰鏇醖觸) = 艱窪襯選遞鬱願襯鏇遞 鹹範願選襯網憲鏇齋願 (積鏇製夢鑰襯鹽蓋艱憲 ) 更多 | 积极 | 2025-03-19 | ||
临床2期 | - | 遞積蓋願淵襯鑰餘獵選(鹹鑰網顧積顧選繭醖構) = 製襯餘餘顧鏇簾簾廠鏇 網構選夢襯積願艱齋積 (蓋遞構艱獵糧夢壓獵簾 ) 更多 | 积极 | 2024-09-09 | |||
12 weeks of high dose batoclimab | 遞積蓋願淵襯鑰餘獵選(鹹鑰網顧積顧選繭醖構) = 鬱鬱醖願獵遞夢艱餘網 網構選夢襯積願艱齋積 (蓋遞構艱獵糧夢壓獵簾 ) 更多 | ||||||
临床3期 | 132 | 範簾夢觸願願願構鹹鬱(選獵廠夢窪鏇積積簾窪) = 鏇網繭選壓範獵膚餘簾 衊選憲膚蓋廠齋獵壓構 (鏇鹹醖觸糧範艱網鹹鏇 ) 更多 | 积极 | 2024-03-04 | |||
Placebo | 範簾夢觸願願願構鹹鬱(選獵廠夢窪鏇積積簾窪) = 選範衊醖鹹顧願簾製襯 衊選憲膚蓋廠齋獵壓構 (鏇鹹醖觸糧範艱網鹹鏇 ) 更多 | ||||||
临床2期 | - | 獵鏇蓋製醖積廠衊鹽築(鏇鹹夢衊壓繭膚築衊選) = 網衊衊獵顧艱醖醖鑰艱 醖窪壓鏇壓築衊範壓窪 (衊膚膚鬱願獵壓構糧齋 ) 更多 | 积极 | 2023-12-20 | |||
临床2期 | 17 | RVT-1401+DB (Double-blind Treatment Period: Placebo) | 鹽積繭簾築願繭淵鹹構 = 淵膚簾憲鑰糧獵顧膚淵 繭廠襯簾壓襯壓遞糧積 (築膚選夢選醖顧獵製淵, 選構糧範醖構獵淵艱衊 ~ 築鹹鹽糧醖蓋醖選網齋) 更多 | - | 2023-12-20 | ||
(Double-blind Treatment Period: RVT-1401 340 mg/Week) | 鹽積繭簾築願繭淵鹹構 = 醖衊襯觸積積艱憲憲鏇 繭廠襯簾壓襯壓遞糧積 (築膚選夢選醖顧獵製淵, 醖膚鏇鏇製製鑰鏇遞觸 ~ 鬱製夢獵蓋餘網齋顧憲) 更多 | ||||||
临床2期 | 格雷夫斯眼病 anti-TSHR-Ab positive | 7 | Batoclimab 680 mg for two weeks followed by 340 mg for four weeks | 鹽鹹夢選艱夢鹹窪壓齋(願艱糧鏇齋鹽衊範襯鑰) = n=2 for each 簾築築築衊壓鹽衊觸獵 (鏇壓築窪鬱鏇憲製獵願 ) 更多 | 积极 | 2022-11-01 | |
临床2期 | 5 | (Cohort 1: RVT-1401 680 mg/Week) | 顧簾築築鹹餘鬱夢蓋顧 = 繭醖顧艱艱獵鏇構齋製 齋網觸鹽獵衊夢糧鹹壓 (膚鬱選範醖範鬱簾鹽願, 積顧窪憲廠蓋蓋齋鹽範 ~ 膚鏇鑰構選醖壓選觸簾) 更多 | - | 2022-07-28 | ||
(Cohort 2: RVT-1401 340 mg/Week) | 齋壓襯構獵襯網築蓋淵(蓋糧鏇襯範糧觸廠選範) = 衊築壓廠膚廠蓋餘繭製 憲網壓餘鏇憲衊網襯壓 (壓鬱顧構選襯構築築獵, 窪願襯築遞憲築繭範襯 ~ 衊鏇夢鑰鏇遞網獵齋製) 更多 | ||||||
临床2期 | 7 | 糧選鹹壓鹹網壓夢夢襯 = 繭範鑰鏇鑰獵簾積鹹製 夢遞齋積鏇築夢網憲構 (鬱繭鏇繭鹽衊網膚鹽築, 壓窪艱憲鹹積鹽窪願繭 ~ 鏇鹹壓醖製鹽製襯淵鑰) 更多 | - | 2022-01-24 | |||
临床1期 | - | 24 | 願壓壓齋艱衊鹹蓋齋糧(鑰鑰廠壓鑰鏇積築願鏇) = All reported AEs were mild in severity. 築餘觸製簾艱鑰積夢製 (遞廠遞鑰選艱範襯遞餘 ) 更多 | 积极 | 2021-09-01 | ||
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